Recent Advances in Molecular and Translational Medicine: Updates in Precision Medicine

Author(s): Usanarat Anurathapan* and Suradej Hongeng

DOI: 10.2174/9789815036756121010015

Hematopoietic Stem Cell Transplant in Inherited Genetic Diseases

Pp: 225-234 (10)

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* (Excluding Mailing and Handling)

  • * (Excluding Mailing and Handling)

Abstract

SHS investigation development is considered from the geographical and historical viewpoint. 3 stages are described. Within Stage 1 the work was carried out in the Department of the Institute of Chemical Physics in Chernogolovka where the scientific discovery had been made. At Stage 2 the interest to SHS arose in different cities and towns of the former USSR. Within Stage 3 SHS entered the international scene. Now SHS processes and products are being studied in more than 50 countries.

Abstract

Hematopoietic stem cell transplant (HSCT) is a curative treatment for many diseases especially inherited genetic diseases such as thalassemia, inherited bone marrow failure syndromes, primary immunodeficiencies, Gaucher disease, Hunter syndrome, or other mucopolysaccharidoses. The two main elements of HSCT are the conditioning regimen and an available donor. The conditioning regimen included combined chemotherapy, monoclonal antibody, or radiation, while the available donor might be HLA-identical or HLA-mismatched.

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